Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

NCT03567460 | Completed

• 1 other identifier: 37176
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise. There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture. For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.

Conditions

Marfan Syndrome

Outcome Measures

Primary Outcomes (1)

• To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.

  The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).

  6 months of intervention

Secondary Outcomes (4)

• To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.

  6 months of intervention

• To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.

  6 months of intervention

• To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.

  6 months of intervention

• To determine if 6 months of regular physical activity improves coping skills in Marfan patients.

  6 months of intervention

Study Arms (1)

10,000 Steps/day for Pediatric Marfan Patients

EXPERIMENTAL

Participants will be given a Garmin VivoFit and asked to take at least 10,000 steps per day. A study coordinator will reach out at least once per week to check in on progress made and help make weekly goals.

Behavioral: Exercise

Interventions

Exercise BEHAVIORAL

Across studies in adolescents in general population, 60 minutes of recommended daily exercise level is achieved, on average, within a total volume of 10,000-11,700 steps. The investigators will assess baseline physical activity quantitatively by using an accelerometer worn on the wrist during waking hours over a 7-day period. Each participant will be given a Garmin device to place on their wrist that will track their steps. Then patients will be asked to complete 10,000 steps daily for 6 months (acclimation over a week), which will be encouraged by daily text messages or e-mail, and weekly phone calls by the intervention team in addition to a Garmin and Facebook peer group.

10,000 Steps/day for Pediatric Marfan Patients

Eligibility Criteria

• Age: 10 Years - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• years of age,
• MFS by revised Ghent criteria,
• Cardiac clearance to exercise by the primary cardiologist.

You may not qualify if:

• Ventricular dysfunction,
• Prior history of aortic surgery.

Sponsors & Collaborators

• Stanford University: lead
• Midwestern University: collaborator

Study Sites (1)

Stanford Children's Health

Palo Alto, California, 94306, United States

Location

Related Publications (1)

• Selamet Tierney ES, Chung S, Stauffer KJ, Brabender J, Collins RT 2nd, Folk R, Li W, Murthy AK, Murphy DJ, Esfandiarei M. Can 10 000 Healthy Steps a Day Slow Aortic Root Dilation in Pediatric Patients With Marfan Syndrome? J Am Heart Assoc. 2022 Dec 6;11(23):e027598. doi: 10.1161/JAHA.122.027598. Epub 2022 Dec 1.

  PMID: 36453629 DERIVED

MeSH Terms

Conditions

Marfan Syndrome

Interventions

Exercise

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Officials

• Seda Tierney, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor in Pediatrics

Study Record Dates

• First Submitted: October 3, 2017
• First Posted: June 25, 2018
• Study Start: January 1, 2017
• Primary Completion: December 31, 2019
• Study Completion: December 31, 2019
• Last Updated: July 27, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)